A Dose-optimization Study of Aldurazyme┬« (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease
Information source: Sanofi
ClinicalTrials.gov processed this data on August 20, 2015
Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Mucopolysaccharidosis I; Hurler's Syndrome; Hurler-Scheie Syndrome; Scheie Syndrome
Intervention: Aldurazyme (Recombinant Human Alpha-L-Iduronidase) (Biological); Aldurazyme (Recombinant Human Alpha-L-Iduronidase) (Biological); Aldurazyme (Recombinant Human Alpha-L-Iduronidase) (Biological); Aldurazyme (Recombinant Human Alpha-L-Iduronidase) (Biological)
Phase: Phase 4
Sponsored by: Genzyme, a Sanofi Company
Official(s) and/or principal investigator(s):
Medical Information, Study Director, Affiliation: Genzyme, a Sanofi Company
The main purpose of this study is to evaluate differences in the pharmacodynamic response of
4 Aldurazyme« (laronidase) dose regimens in patients with Mucopolysaccharidosis I (MPS I).
Official title: A Multicenter, Multinational, Randomized, Dose-Optimization Study of the Safety and Pharmacodynamic Response of Aldurazyme« (Laronidase) in Patients With Mucopolysaccharidosis I
Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Primary outcome: Percent Change From Baseline to Week 26 in Urinary Glycosaminoglycan (GAG) Level
Percent Change From Baseline to Week 26 in Liver Organ Volume
Change From Baseline to Week 26 in Six Minute Walk Test (6MWT)
Minimum age: N/A.
Maximum age: N/A.
- Have a documented diagnosis of MPS I, confirmed by measurable clinical signs and
symptoms of MPS I, and a documented fibroblast or leukocyte ╬▒-L-iduronidase enzyme
activity level of less than 10% of the lower limit of the normal range of the
- Weigh at least 12. 5 kg.
- Have serum creatinine and blood urea nitrogen (BUN) values within age appropriate
- Provide signed, written informed consent prior to any protocol-related procedures
being performed. Consent of a legally authorized guardian(s) is (are) required for
patients under 18 years. If the patient is under 18 years old and can understand the
consent, written informed consent will be required from both the patient and the
- Have previously received Aldurazyme┬« (laronidase).
- Have a suspected hypersensitivity to Aldurazyme┬« (laronidase) or known
hypersensitivity to components of the infusion solution.
- Have previously undergone hematopoietic stem cell transplantation (HSCT; i. e., from
bone marrow [BMT], peripheral blood, or umbilical cord blood) or other major organ
- Have a medical condition, serious inter-current illness, or other extenuating
circumstance that may interfere with study compliance including all prescribed
evaluations and follow-up activities, except the 6MWT. (Note: All patients may not be
capable of performing the 6MWT due to age and/or maturity level. Exemption from
performing the 6MWT must be obtained in writing by the investigator from the
sponsor's medical monitor prior to enrollment).
- Have an acute illness that requires surgical intervention, and/or anticipates surgery
during study participation, and/or has had surgery within 30 days prior to study
- Have received an investigational drug within 30 days prior to study enrollment.
- Is pregnant or lactating. Female patients of childbearing potential must have a
negative pregnancy test [urine ╬▓-human chronic gonadotropin (hCG)] at entry (prior to
the first infusion). Note: All female patients of childbearing potential and
sexually mature males must be advised to use a medically accepted method of
contraception throughout the study.
Locations and Contacts
Universidade Federal de Minas Gerais, Belo Horizonte CEP 30130-100, Brazil
Hospital de Clinical de Porto Alegre, Porto Alegre CEP 90035-003, Brazil
Universidade Federal de Sao Paulo, San Paulo CEP 04023-062, Brazil
Division of Clinical and Metabolic Genetics, Toronto, Ontario M5G 1X8, Canada
Hospital Infantil Joana de Gusmao, Florianopolis, Santa Catarina CEP 88025-601, Brazil
Starting date: December 2004
Last updated: March 17, 2015